American Association for Cancer Research
mct-23-0094_supplementary_figure_10_suppsf10.docx (3.07 MB)

Supplementary Figure 10 from Elimusertib has Antitumor Activity in Preclinical Patient-Derived Pediatric Solid Tumor Models

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journal contribution
posted on 2024-04-02, 07:21 authored by Fabian F. Pusch, Heathcliff Dorado García, Robin Xu, Dennis Gürgen, Yi Bei, Lotte Brückner, Claudia Röefzaad, Jennifer von Stebut, Victor Bardinet, Rocío Chamorro Gonzalez, Angelika Eggert, Johannes H. Schulte, Patrick Hundsdörfer, Georg Seifert, Kerstin Haase, Beat W. Schäfer, Marco Wachtel, Anja A. Kühl, Michael V. Ortiz, Antje M. Wengner, Monika Scheer, Anton G. Henssen

Supplementary Figure 10 shows the IHC markers tested in MNA and NMNA NB PDXs.


Deutschen Konsortium für Translationale Krebsforschung (DKTK)

BIH-Charité Clinician Scientist Program

Deutsche Krebshilfe (German Cancer Aid)

Pharmaceuticals Bayer

'la Caixa' Foundation ('la Caixa')

Deutsche Forschungsgemeinschaft (DFG)

Wilhelm Sander-Stiftung (Wilhelm Sander Foundation)

HORIZON EUROPE Reforming and enhancing the European Research and Innovation system (REERIS)

Charité 3R, Charité - Universitätsmedizin Berlin



The small-molecule inhibitor of ataxia telangiectasia and Rad3-related protein (ATR), elimusertib, is currently being tested clinically in various cancer entities in adults and children. Its preclinical antitumor activity in pediatric malignancies, however, is largely unknown. We here assessed the preclinical activity of elimusertib in 38 cell lines and 32 patient-derived xenograft (PDX) models derived from common pediatric solid tumor entities. Detailed in vitro and in vivo molecular characterization of the treated models enabled the evaluation of response biomarkers. Pronounced objective response rates were observed for elimusertib monotherapy in PDX, when treated with a regimen currently used in clinical trials. Strikingly, elimusertib showed stronger antitumor effects than some standard-of-care chemotherapies, particularly in alveolar rhabdomysarcoma PDX. Thus, elimusertib has strong preclinical antitumor activity in pediatric solid tumor models, which may translate to clinically meaningful responses in patients.

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